Dear doctors,

On behalf of the Board of Directors of the Thalassaemia International Federation (TIF), it is my great privilege to write the foreword to the 4th edition of the Guidelines for the Management of Transfusion-Dependent Thalassaemia (TDT).

One can today, and this has been so for some time now, safely state that haemoglobin disorders especially thalassaemia – characterised in the very early years and perhaps until the late 1970s as a fatal childhood disease – can be effectively prevented and appropriately managed.

Published data on increased survival, reduced annual number of deaths and morbidity rates coupled with improved quality of life and social integration demonstrated beyond any doubt that the fight to address and effectively control this disorder has nearly been won. Particularly if one includes in the equation the dramatic progress achieved in the last few years in the development and authorisation of two long awaited innovative therapies and in strengthening research on many other drugs today in the pipeline at different stages of development.

Sadly however, such progress has occurred only in countries, mainly with high disease prevalence, where national health and social care systems are providing universal coverage to their populations including importantly patients with chronic diseases, and where robust medical/public health, haematology and paediatric infrastructures existed to support the development of disease-specific services.

Unfortunately however, this picture is seen only in a few countries mainly with developed economies and the naked truth is that of these huge medical and scientific advances happening through the years, only a very small portion of the global patient population benefits and overall this is estimated by TIF to be well less than 20%.

It is evident that countries, mainly with high disease prevalence, need to be further empowered to recognise the value of strengthening disease-specific policies of prevention and health/social care of patients with these disorders. The World Health Organization (WHO) through its disease-specific resolutions – but also a number of other relevant resolutions and programmes, including Blood Safety, Patient Safety, Congenital Anomalies and Birth Defects, Human Genomics in Global Health, Access to medicines and health products just to name a few - can provide considerable support to national efforts. In addition, the work of other regional health fora and initiatives can contribute to national actions; And in this context the European Union’s (EU) work on Rare Diseases since the 1990s, is indeed exemplary and many related recommendations as well as directives and regulations give food for thought and provide a basis for others beyond the EU on which to build. As countries become more and more engaged in promoting the UN 2030 SDGs (Sustainable Development Goals), it is hoped that progress in this field, albeit to a different extent across countries and regions of the world, will be achieved.

TIF remains committed to continuing to support, with its work, the national patients’/parents’ organisations, the health care professionals’ community and the competent authorities. The preparation, publication, translation and free distribution across countries of Guidelines, an important deliverable of its educational programme, has been widely recognised as a significant contribution to the care of patients with these disorders.

Their adoption by competent health authorities and/or medical bodies as published, or as content on which to build national Guidelines, has been a most valuable tool in the education and/or training of treating physicians across the world, and is one of TIF’s activities considered to have the highest measurable impact on the health and lives of patients with thalassaemia across the globe.

National Thalassaemia patients/parents associations, members of TIF, across the world have contributed significantly to the work of TIF and have made a true and impressive difference with their voluntary work towards national improvements, complementing and on occasions a undertaking Governments’ commitments. To this end therefore, TIF poses a humble but clear request to Governments all over the world for their highly needed support.

The engagement and contribution of patient advocates in national, regional and international efforts in promoting progress for the control and care of thalassaemia are indeed invaluable elements. TIF has been fighting since its establishment in 1986 to bring to the forefront the value of the meaningful involvement of patients at the decision-making level. This updated 4th edition of the Guidelines for the Management of Transfusion-Dependent Thalassaemia proudly incorporates an additional chapter dedicated to the value of patient engagement. This chapter aims to cover comprehensively this topic, underscoring the progress that has been achieved through the years in ‘making’ patients “expert” advocates of their needs as well as the many and multiple benefits of their meaningful involvement in decision making at national, regional and international level.

The huge economic, social and health challenges that have arisen as a result of the recent COVID-19 pandemic, have undoubtedly added to the already difficult and challenging conditions of many countries across the world with regard to the care of these disorders. The significant contradiction here is that parallel to the pandemic tragedy, research and science presented to the global thalassaemia community, patients and health care professionals alike, an invaluable and long awaited gift: the completion of clinical trials and the authorisation by the FDA (Food and Drug Administration) and the EMA (European Medicines Agency) in 2019 and 2020 of two innovative products for the care and cure of thalassaemia. It is thus more than ever essential for every involved stakeholder in the care of patients, Governments, health care professionals and very importantly patients and parents themselves through their associations, to become more actively and productively engaged in the strengthening of those tools, policies and collaborations that would ensure the access of patients to such medical advancements. At the same time and very importantly, all of us jointly should safeguard carefully the existing prevention and disease-specific policies, medical and public health infrastructures including transfusion services, against any regression, threat or risk, in the name of the pandemic, that would jeopardise the quality and/or standard of care and services a country has achieved for these patients through the years.

TIF remains committed to continuing and strengthening its support to the patients it represents through their national associations and its collaboration with decision-making bodies, health care professionals, industry and other relevant stakeholders at national and international level.

My very special and humble request on behalf of the Board of Directors of the Thalassaemia International Federation, through this foreword to every treating professional studying these Guidelines, is to make every effort with his/her national competent authority to support the development and/or strengthening of those policies and programmes that would allow the adoption and implementation at national level of these valuable and evidenced-based recommendations made by international medical/scientific experts.

In this context, allow me on behalf of the Board, to express my deepest and most sincere gratitude to each one of the key authors, the reviewers/editors and to every contributing author of this book, without the work of whom, these Guidelines as well as any other publications and educational activity of TIF would not have been possible to deliver.

Finally, taking this opportunity, the Board of Directors of TIF, wishes to dedicate this 4th edition of the Guidelines to those two medical specialists and researchers who are no longer with us and whom we lost in recent years but who, with their work and devotion, have made history and set milestones in this field – to Sir David John Weatherall and to Professor George Stamatoyannopoulos.

On behalf of the Board of Directors